The FDA Approval Process: Can We Have This Drug Now?

Questions and answers about the FDA and its process for approving drugs

Article Highlights:
  • The U.S. Food and Drug Administration (FDA) usually requires that investigational drugs go through laboratory development followed by phase 1, phase 2 and phase 3 human trials, but there are mechanisms that the FDA has created to speed up the process when a drug is being developed for a serious or life-threatening disorder.
  • Fast track, breakthrough therapy and priority review are designations that accelerate the process the FDA uses to review applications for new drugs.
  • Accelerated approval and expanded access are mechanisms that the FDA uses to allow patients to receive medications before they have received full approval from the agency.
  • To learn more about the expanded access process, read Four Perspectives on Expanded Access, where four professionals share their thoughts about access to drugs that have not gone through the full approval process.
by Margaret Wahl on July 7, 2014 - 9:19am

Quest Summer 2014

To people faced with life-threatening diseases, the U.S. Food and Drug Administration (FDA) can seem like an indifferent obstacle, keeping them from treatments that would otherwise be available. But the reality is much more layered and complex.

Here, MDA answers some frequently asked questions about how the FDA works to shed light on this topic. 

Q: Why does the FDA have stringent regulations regarding the safety and efficacy (effectiveness) of drugs?

A: The FDA’s goal is to protect people from unsafe or ineffective drugs. A short glance at history reveals a lot about why the FDA came into being in 1930 and has survived into the 21st century. Two examples stand out — the deaths in the late 1930s of more than 100 people who took an infection-fighting drug that was unfortunately dissolved in a deadly poison, and the birth in the 1960s of babies with limb deformities after their mothers took what seemed to be a harmless pill (thalidomide) to treat their pregnancy-associated nausea. 

And throughout history, vulnerable patients have been lured by expensive treatments that may or may not have harmed them but resulted in serious financial setbacks or kept them from seeking other, possibly helpful, remedies.

The FDA has several mechanisms it can use to speed up the review of applications for new drugs or allow patients access to new drugs before they are fully approved. Click on the illustration for an enlarged version.

It is from this history that the FDA has developed its current standards of safety and efficacy for medicines and medical devices.

Q: Does the FDA realize there are individuals and families who are counting on new drugs to save their lives? Can the review and approval process be made faster when lives are at stake?

A: Yes. The FDA has mechanisms for speeding up the process it uses to review developers’ applications for new drugs. These include “fast track,” “breakthrough therapy” and “priority review.” In addition, the FDA can supply an investigational drug to patients through its “expanded access” mechanism and can allow access to a drug that is not fully approved through its “accelerated approval” pathway. (For more, see Speeding Things Up on this page.)

Q: Can the FDA alone provide access to an unapproved drug?

A: No. The FDA can provide a legal pathway for access to a drug that is not fully approved, but it cannot make the company behind that drug supply it.

Q: What are the risks to a company of supplying a drug that has not gone through the full approval process?

A: According to drug companies, supplying a drug to patients before it is fully approved can be very risky, especially for a small firm with limited resources. Companies depend on having a fully approved product on the market or at least a clear path laid out for that goal. That’s how they attract investors, and that’s how they stay in business. 

When supplying a medication that the FDA has not fully approved, the developer of the medication runs several risks, including:

  • not being able to successfully conduct clinical trials required for approval, since patients have the opportunity to obtain the drug without participating in a trial;
  • difficulty meeting high demand for a limited supply of the drug;
  • not having the necessary funds to take the drug through the approval process because so much money had to be spent on manufacturing and distribution for an expanded access program; and
  • not being able to carry out the necessary trials needed for full approval because of adverse events that may have occurred in patients taking the unapproved drug (although the FDA says it knows of no such examples).

Q: What are the risks to patients taking a drug that has not been approved or has been approved through an accelerated approval pending confirmatory studies?

A: To the family of a loved one facing a fatal disease, all risks associated with unproven treatments may seem worth taking. Unfortunately, many people who try unproven drugs actually get worse.

Q: Are there ways that this process could be improved?

A: Arthur Caplan, a bioethicist based at New York University, believes the process could be improved by developing a standard template that patients and families could use to apply for access to unapproved drugs and establishing an independent advisory board consisting of patient advocates, scientists and ethicists to review such requests. The board, Caplan proposes, might help small companies get over the financial hurdles associated with providing an experimental drug to patients by supplying funding derived from taxes on the industry or from government funding.       

Eteplirsen is an experimental “exon-skipping” drug designed to treat about 13 percent of patients with Duchenne muscular dystrophy (DMD) — those with mutations in the dystrophin gene that are near exon 51 and who can potentially be helped by having the genetic instructions for this section “skipped” during the process muscle cells use to make dystrophin protein. MDA has supported the development of exon skipping for DMD since the 1990s.

A 12-participant trial has shown that eteplirsen appears to stabilize walking ability over more than two years, although two participants stopped walking very early in this study and were not included in walking test analyses. The drug appears to be safe and well-tolerated.

In April 2014, the drug’s developer, Sarepta Therapeutics, announced it planned to submit a new drug application to the FDA for eteplirsen by the end of 2014, using the agency’s accelerated approval mechanism. If accelerated approval is granted, the company would still need to conduct a confirmatory study. However, in the meantime, patients would have access to the drug. Sarepta plans to begin a confirmatory trial of eteplirsen during 2014.

As this article was going to press, Dutch biotechnology company Prosensa announced that it will submit an accelerated approval application for its DMD exon-skipping drug, drisapersen.

To learn more about eteplirsen and drisapersen, see Exon-Skipping Drugs Forge Ahead.

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